CRISPR-Edited Allogeneic Anti-CD19 CAR-T Cell Therapy, in Patients With Refractory Systemic Lupus Erythematosus (GALLOP)

Caribou Biosciences

Status and phase: Withdrawn; Phase 1

Conditions: Lupus Nephritis; Lupus; Systemic Lupus Erythematosus; Lupus Erythematosus

Treatments: Drug: CB-010

Study type: Interventional

Funder types: Industry

Identifiers

NCT06752876

CB10LUPA

Details and patient eligibility

About

This is a Phase 1 study to evaluate the safety and efficacy of a single infusion of CB-010 in patients with refractory Systemic Lupus Erythematosus (SLE) with cohorts for lupus nephritis (LN) and extrarenal lupus (ERL).

Full description

Participants enrolled can expect to be on the study for a total duration of approximately 2 years, during which there will be a screening period followed by a single administration of CB-010 and then 24 months of safety follow-up and monitoring.

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Clinical diagnosis of SLE according to 2019 EULAR/ACR classification criteria for at least 6 months

• Cohort 1 LN:

  1. Class III or IV lupus nephritis
  2. Urine protein-to-creatinine ratio (UPCR) ≥ 0.8 mg/mg
  3. Refractory to glucocorticoids and at least 2 immunosuppressive therapies

• Cohort 2 ERL (Patients with class I and II LN may be included in the ERL cohort if their SLEDAI-2K is ≥ 8):

  1. SLEDAI-2K ≥ 8
  2. Refractory to glucocorticoids, and at least 2 immunosuppressive therapies

• Adequate renal, hepatic, pulmonary, and cardiac function, with specific laboratory criteria

• Females must be either of nonchildbearing potential, defined as postmenopausal or surgically sterile or agree to use a highly effective double barrier method of contraception or vasectomized partner

Exclusion criteria

• Has active severe central nervous system (CNS) lupus in the previous 3 months from planned LD start date
• Has received any other investigational treatment for any indication within the 4 weeks or 5 half-lives
• Prior treatment with cellular therapy (genetically modified cells), gene therapy directed at any target, allogenic or autologous stem cell transplant or organ transplant
• History of infection with human immunodeficiency virus or evidence of hepatitis B or C virus infection
• History of hypersensitivity to Cyclophosphamide, Fludarabine, or any of the components of CB-010
• Received a live vaccine ≤ 6 weeks prior to start of LD
• Patients for whom magnetic resonance imaging (MRI) studies are contraindicated or who cannot tolerate them

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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0 participants in 2 patient groups

Cohort 1 Lupus Nephritis (LN)

Experimental group

Treatment:

Drug: CB-010

Cohort 2 Extrarenal Lupus (ERL)

Experimental group

Treatment:

Drug: CB-010